CN112020561A - 用于治疗人受试者中非年龄相关的听力损害的组合物和方法 - Google Patents

用于治疗人受试者中非年龄相关的听力损害的组合物和方法 Download PDF

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CN112020561A
CN112020561A CN201980026498.XA CN201980026498A CN112020561A CN 112020561 A CN112020561 A CN 112020561A CN 201980026498 A CN201980026498 A CN 201980026498A CN 112020561 A CN112020561 A CN 112020561A
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amino acids
amino acid
acid substitutions
amino
acids
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E.J.西蒙斯
R.吴
E.雷辛格
H.阿尔-莫耶德
S.库格勒
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Akus Co ltd
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Akus Co ltd
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CN201980026498.XA 2018-02-22 2019-02-22 用于治疗人受试者中非年龄相关的听力损害的组合物和方法 Pending CN112020561A (zh)

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US201862634088P 2018-02-22 2018-02-22
US62/634,088 2018-02-22
PCT/US2019/019268 WO2019165292A1 (en) 2018-02-22 2019-02-22 Compositions and methods for treating non-age-associated hearing impairment in a human subject

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US (2) US20200390905A1 (https=)
EP (1) EP3755805A4 (https=)
JP (2) JP7408228B2 (https=)
KR (2) KR20260015297A (https=)
CN (1) CN112020561A (https=)
AU (2) AU2019224121B2 (https=)
BR (1) BR112020016919A2 (https=)
CA (1) CA3091676A1 (https=)
CL (1) CL2020002161A1 (https=)
EA (1) EA202091995A1 (https=)
IL (2) IL325122A (https=)
MA (1) MA51918A (https=)
MX (2) MX2020008763A (https=)
SG (1) SG11202007491UA (https=)
WO (1) WO2019165292A1 (https=)

Cited By (7)

* Cited by examiner, † Cited by third party
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CN113063944A (zh) * 2021-03-05 2021-07-02 李朴 血清gsdme在b淋巴细胞白血病诊断、疗效监测及预后评估上的用途
CN113337507A (zh) * 2021-08-05 2021-09-03 首都医科大学附属北京友谊医院 Otof 1273 (C>T)基因定点突变的耳聋小鼠模型的构建方法及其应用
CN114807224A (zh) * 2022-04-25 2022-07-29 中国人民解放军军事科学院军事医学研究院 一种可视化斑马鱼胆汁酸代谢模型的构建方法及其应用
CN114854762A (zh) * 2022-06-09 2022-08-05 上海鼎新基因科技有限公司 编码人otof的核酸序列及其应用
CN115141896A (zh) * 2022-09-04 2022-10-04 上海鼎新基因科技有限公司 一种AAV组织分布的qPCR检测方法
CN116925239A (zh) * 2023-07-17 2023-10-24 苏州星奥拓维生物技术有限公司 双载体系统表达Otof基因的组合物和方法
CN117305367A (zh) * 2023-08-21 2023-12-29 复旦大学附属眼耳鼻喉科医院 一种表达全长耳畸蛋白的双aav载体系统及其应用

Families Citing this family (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3510161A4 (en) 2016-08-23 2020-04-22 Akouos, Inc. COMPOSITIONS AND METHODS FOR TREATING PERSONAL HEARING LOSS IN A PERSON
KR102606810B1 (ko) 2017-05-05 2023-11-27 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 오토펄린을 발현시키기 위한 조성물 및 방법
US11660353B2 (en) 2018-04-27 2023-05-30 Decibel Therapeutics, Inc. Compositions and methods for treating sensorineural hearing loss using otoferlin dual vector systems
CN112703016B (zh) 2018-04-27 2024-11-22 分贝治疗公司 肌球蛋白15启动子及其用途
EP4295913A3 (en) * 2019-01-18 2024-03-06 Institut Pasteur Aav-mediated gene therapy restoring the otoferlin gene
CA3129422A1 (en) 2019-02-08 2020-08-13 Decibel Therapeutics, Inc. Myosin 15 promoters and uses thereof
EP3921032A4 (en) 2019-02-08 2022-11-30 Decibel Therapeutics, Inc. COMPOSITIONS AND METHODS FOR TREATMENT OF SENSORINEURAL HEARING LOSS USING OTOFERLIN DOUBLE VECTOR SYSTEMS
EP4007814A1 (en) 2019-07-26 2022-06-08 Akouos, Inc. Methods of treating hearing loss using a secreted target protein
KR20220130093A (ko) 2019-10-30 2022-09-26 데시벨 테라퓨틱스, 인크. 오토펄린 듀얼 벡터 시스템을 사용한 감각신경성 난청을 치료하기 위한 조성물 및 방법
KR20220157944A (ko) 2020-02-21 2022-11-29 아카우오스, 인크. 인간 대상체에서 비-연령-연관 청각 장애를 치료하기 위한 조성물 및 방법
CN115666658A (zh) 2020-04-01 2023-01-31 佛罗里达州大学研究基金会 具有提高的治疗ush1b的安全性的双aav-myo7a载体
IL298128A (en) 2020-05-13 2023-01-01 Akouos Inc Compositions and methods for treating slc26a4-associated hearing loss
IL304006A (en) 2020-12-29 2023-08-01 Akouos Inc Compositions and methods for treating hearing loss and/or vision loss associated with CLRN1
EP4294460A4 (en) * 2021-02-19 2025-06-25 Decibel Therapeutics, Inc. Methods for treating sensorineural hearing loss using otoferlin dual vector systems
WO2023036966A1 (en) * 2021-09-10 2023-03-16 Institut Pasteur Dual recombinant aav8 vector system encoding isoform 5 of otoferlin and uses thereof

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2001070972A2 (en) * 2000-03-24 2001-09-27 Yasunaga, Shin'ichiro Multiple human and mouse otoferlin isoforms
US20160076054A1 (en) * 2013-04-18 2016-03-17 Fondazione Telethon Effective delivery of large genes by dual aav vectors

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8173792B2 (en) * 2007-02-09 2012-05-08 The Board Of Trustees Of The Leland Stanford Junior University Method for regulating protein function in cells using synthetic small molecules
HK1220488A1 (zh) * 2013-03-15 2017-05-05 The Children's Hospital Of Philadelphia 含有填充者/填充物多核苷酸序列的载体及其使用方法
SI3265571T1 (sl) * 2015-03-03 2022-10-28 Fondazione Telethon Več vektorski sistem in njegove uporabe
EP3510161A4 (en) * 2016-08-23 2020-04-22 Akouos, Inc. COMPOSITIONS AND METHODS FOR TREATING PERSONAL HEARING LOSS IN A PERSON
KR102606810B1 (ko) 2017-05-05 2023-11-27 유니버시티 오브 플로리다 리서치 파운데이션, 인코포레이티드 오토펄린을 발현시키기 위한 조성물 및 방법
KR20220157944A (ko) * 2020-02-21 2022-11-29 아카우오스, 인크. 인간 대상체에서 비-연령-연관 청각 장애를 치료하기 위한 조성물 및 방법

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2001070972A2 (en) * 2000-03-24 2001-09-27 Yasunaga, Shin'ichiro Multiple human and mouse otoferlin isoforms
US20160076054A1 (en) * 2013-04-18 2016-03-17 Fondazione Telethon Effective delivery of large genes by dual aav vectors

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
ALEMI,AURASH: "AOS research grant: restoration of hearing in the otoferlin knockout mouse using viral gene therapy" *
BUDENZ, CAMERON L. ET AL.: "Differential Effects of AAV.BDNF and AAV.Ntf3 in the Deafened Adult Guinea Pig Ea" *

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* Cited by examiner, † Cited by third party
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CN113337507A (zh) * 2021-08-05 2021-09-03 首都医科大学附属北京友谊医院 Otof 1273 (C>T)基因定点突变的耳聋小鼠模型的构建方法及其应用
CN113337507B (zh) * 2021-08-05 2021-11-12 首都医科大学附属北京友谊医院 Otof 1273 (C>T)基因定点突变的耳聋小鼠模型的构建方法及其应用
CN114807224A (zh) * 2022-04-25 2022-07-29 中国人民解放军军事科学院军事医学研究院 一种可视化斑马鱼胆汁酸代谢模型的构建方法及其应用
CN114807224B (zh) * 2022-04-25 2023-09-29 中国人民解放军军事科学院军事医学研究院 一种可视化斑马鱼胆汁酸代谢模型的构建方法及其应用
CN114854762A (zh) * 2022-06-09 2022-08-05 上海鼎新基因科技有限公司 编码人otof的核酸序列及其应用
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WO2025040112A1 (zh) * 2023-08-21 2025-02-27 复旦大学附属眼耳鼻喉科医院 一种表达全长耳畸蛋白的双aav载体系统及其应用

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